Fabry DiseaseMOSNCT06941025
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
An observational study of Fabry Disease and Pregnancy, run by Chiesi Farmaceutici S.p.A. at 5 sites in 5 countries.
Trial summary
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Fabry Disease, Pregnancy, Pregnancy Complications
- What is being tested?
- Not applicable- observational study
- How many people
- 10 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 23 May 2025to Dec 2034
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Pregnant and/or breastfeeding women with Fabry disease and their infants, after exposure to at least 1 dose of pegunigalsidase alfa during pregnancy and/or during lactation.
Who may be able to join
- Female patients with Fabry disease who have been exposed to at least 1 dose of pegunigalsidase alfa at any time during pregnancy (defined as having received pegunigalsidase alfa within 30 days prior to the DOC and/or during pregnancy) and/or during lactation, and their infants.
- o DOC, defined as 20/7 gestational weeks, will be calculated from last menstrual period [LMP] or ultrasound
- Patient or parent/legally authorized representative must be able to understand and provide consent through an Institutional Review Board / Independent Ethics Committee (IRB/IEC) approved Informed Consent Form.
Who cannot join
- None
Trial locations and contacts
5 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 5 countries
United States1 site, 1 recruiting
- Recruiting
No physical study sites - Decentralized, web-based registry
Washington D.C., District of Columbia, 20001
Germany1 site, 1 recruiting
- Recruiting
No physical study sites - Decentralized, web-based registry
Berlin
Italy1 site, 1 recruiting
- Recruiting
No physical study sites - Decentralized, web-based registry
Rome
Spain1 site, 1 recruiting
- Recruiting
No physical study sites - Decentralized, web-based registry
Madrid
United Kingdom1 site, 1 recruiting
- Not yet recruiting
No physical study sites - Decentralized, web-based registry
London
About this study
Official title: Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj/Pegunigalsidase Alfa) During Pregnancy and/or Lactation
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation.
The main objectives are to:
Read more
- Assess pregnancy outcomes, including maternal and infant health.
- Evaluate the occurrence of congenital malformations and other neonatal outcomes.
This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs).
Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.
Read the detailed description
This is a global, decentralized, single-arm, prospective and retrospective observational registry designed to evaluate pregnancy and infant outcomes in women with Fabry disease who have been exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The registry aims to assess maternal and infant safety, pregnancy outcomes, and the occurrence of congenital malformations and other neonatal conditions.
The registry will enroll patients over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data collection will be facilitated through a secure, centralized web-based platform, where patients and physicians can enter information using electronic case report forms (eCRFs).
Enrollment \& Data Collection:
Patients can be enrolled at any time, either during pregnancy or after delivery. Depending on the timing of enrollment, data will be collected retrospectively and/or prospectively.
Pregnancy and clinical outcomes will be monitored from enrollment until the infant reaches 12 months of age.
Collected data includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health parameters.
Reported congenital malformations will be classified according to established criteria (e.g., MACDP, EUROCAT) and adjudicated by an independent Scientific Advisory Committee.
The registry is observational and does not alter clinical care, physician treatment decisions, or patient management.
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Being tested
- Not applicable- observational study
- Medical subject headings
- Fabry Disease, Pregnancy Complications
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Not applicable- observational studyOther
Not applicable - observational study
| Study group | Role | What they receive |
|---|---|---|
| Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation | Not described Receives: Other: Not applicable- observational study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pregnancy outcome: Number of live births
Measured: at the delivery, after an average of 40 weeks of pregnancy
Pregnancy outcome: Number of preterm birth
Measured: at delivery, prior to 37 weeks of gestation
Pregnancy outcome: Number of pregnancy losses (number of spontaneous abortions , number of pregnancy terminations, number of foetal deaths or stillbirths)
Measured: spontaneous abortions: up to 20 weeks of pregnancy; pregnancy terminations: through the pregnancy; number of foetal deaths or stillbirths: greater than 20 weeks of pregnancy and through the pregnancy, average of 40 weeks
Secondary outcomes (16)
Number of neonates/infants with MCMs
Measured: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of ectopic or molar pregnancies
Measured: through the pregnancy, an average of 40 weeks
Number of women with obstetric and delivery complications
Measured: at the delivery, an average of 40 weeks of pregnancy
Number of women with complications of preeclampsia or eclampsia
Measured: through the pregnancy, an average of 40 weeks
Number of women with complications of preterm prelabour rupture of membrane
Measured: at delivery, prior to 37 weeks of gestation
Number of neonates/infants with minor congenital malformations
Measured: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of infants with developmental deficiency
Measured: up to 12 months of infant age
Number of hospitalisations in infants
Measured: up to 12 months of infant age
Mortality in infants, including neonatal death and infant death
Measured: up to 12 months of infant age
Head circumference in infants (cm)
Measured: up to 12 months of infant age
Weight in infants (kilograms)
Measured: up to 12 months of infant age
Length in infants (cm)
Measured: up to 12 months of infant age
Number of infants born as SGA
Measured: up to 12 months of infant age
Number of infants with postnatal growth deficiency or FTT
Measured: up to 12 months of infant age
Duration of breastfeeding, number of exclusively breastfeeding women and number of breastfeeding women supplemented with formula
Measured: up to 12 months of infant age
Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding
Measured: up to 12 months of infant age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2025
- Study start23 May 2025
- Primary completionDec 2034 (estimated)
- Study completionDec 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06941025
- Sponsor's protocol ID
- CLI-06657AA1-06
- Acronym
- MOS
- Status last verified
- Mar 2026
- First submitted
- 16 Apr 2025
- Last updated
- 19 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj/Pegunigalsidase Alfa) During Pregnancy and/or Lactation
- ClinicalTrials.gov ID
- NCT06941025
- Acronym / sponsor ID
- MOS / CLI-06657AA1-06
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- 23 May 2025 (actual)
- Primary completion
- Dec 2034 (estimated)
- Study completion
- Dec 2034 (estimated)
- First posted
- 23 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation.
The main objectives are to:
- Assess pregnancy outcomes, including maternal and infant health.
- Evaluate the occurrence of congenital malformations and other neonatal outcomes.
This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs).
Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.
Detailed description
This is a global, decentralized, single-arm, prospective and retrospective observational registry designed to evaluate pregnancy and infant outcomes in women with Fabry disease who have been exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The registry aims to assess maternal and infant safety, pregnancy outcomes, and the occurrence of congenital malformations and other neonatal conditions.
The registry will enroll patients over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data collection will be facilitated through a secure, centralized web-based platform, where patients and physicians can enter information using electronic case report forms (eCRFs).
Enrollment \& Data Collection:
Patients can be enrolled at any time, either during pregnancy or after delivery. Depending on the timing of enrollment, data will be collected retrospectively and/or prospectively.
Pregnancy and clinical outcomes will be monitored from enrollment until the infant reaches 12 months of age.
Collected data includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health parameters.
Reported congenital malformations will be classified according to established criteria (e.g., MACDP, EUROCAT) and adjudicated by an independent Scientific Advisory Committee.
The registry is observational and does not alter clinical care, physician treatment decisions, or patient management.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation | – | Other: Not applicable- observational study |
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Pregnant and/or breastfeeding women with Fabry disease and their infants, after exposure to at least 1 dose of pegunigalsidase alfa during pregnancy and/or during lactation.
Key inclusion criteria (3 listed)
- Female patients with Fabry disease who have been exposed to at least 1 dose of pegunigalsidase alfa at any time during pregnancy (defined as having received pegunigalsidase alfa within 30 days prior to the DOC and/or during pregnancy) and/or during lactation, and their infants.
- o DOC, defined as 20/7 gestational weeks, will be calculated from last menstrual period [LMP] or ultrasound
- Patient or parent/legally authorized representative must be able to understand and provide consent through an Institutional Review Board / Independent Ethics Committee (IRB/IEC) approved Informed Consent Form.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Not applicable- observational studyNot applicable - observational study | Other | Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation |
Primary outcome
Pregnancy outcome: Number of live births
Time frame: at the delivery, after an average of 40 weeks of pregnancy
Pregnancy outcome: Number of preterm birth
Time frame: at delivery, prior to 37 weeks of gestation
Pregnancy outcome: Number of pregnancy losses (number of spontaneous abortions , number of pregnancy terminations, number of foetal deaths or stillbirths)
Time frame: spontaneous abortions: up to 20 weeks of pregnancy; pregnancy terminations: through the pregnancy; number of foetal deaths or stillbirths: greater than 20 weeks of pregnancy and through the pregnancy, average of 40 weeks
16 secondary outcomes
Number of neonates/infants with MCMs
Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of ectopic or molar pregnancies
Time frame: through the pregnancy, an average of 40 weeks
Number of women with obstetric and delivery complications
Time frame: at the delivery, an average of 40 weeks of pregnancy
Number of women with complications of preeclampsia or eclampsia
Time frame: through the pregnancy, an average of 40 weeks
Number of women with complications of preterm prelabour rupture of membrane
Time frame: at delivery, prior to 37 weeks of gestation
Number of neonates/infants with minor congenital malformations
Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of infants with developmental deficiency
Time frame: up to 12 months of infant age
Number of hospitalisations in infants
Time frame: up to 12 months of infant age
Mortality in infants, including neonatal death and infant death
Time frame: up to 12 months of infant age
Head circumference in infants (cm)
Time frame: up to 12 months of infant age
Weight in infants (kilograms)
Time frame: up to 12 months of infant age
Length in infants (cm)
Time frame: up to 12 months of infant age
Number of infants born as SGA
Time frame: up to 12 months of infant age
Number of infants with postnatal growth deficiency or FTT
Time frame: up to 12 months of infant age
Duration of breastfeeding, number of exclusively breastfeeding women and number of breastfeeding women supplemented with formula
Time frame: up to 12 months of infant age
Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding
Time frame: up to 12 months of infant age
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2026.
- Official registry website with information on eligibility, participation, and data collection for the observational study on pregnancy and infant outcomes in women with Fabry disease.Link provided by the sponsor.
- Official registry website with information on eligibility, participation, and data collection for the observational study on pregnancy and infant outcomes in women with Fabry disease.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease and Pregnancy, run by Chiesi Farmaceutici S.p.A. at 5 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Germany, Italy, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chiesi Clinical Trial (+3905212791, clinicaltrials_info@chiesi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pregnancy outcome: Number of live births (time frame: at the delivery, after an average of 40 weeks of pregnancy). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2026.